Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening ... Read more
Updates
-
-
Stem Cell Therapy Considered Safe and Eases Symptoms in Adults With RDEB, Phase 1/2 Trial Shows
Using mesenchymal stem cells to treat adults with recessive dystrophic epidermolysis bullosa (RDEB) appears to be safe and may ease symptoms such as itching ... Read more -
On Loving Yourself First
What better topic could there be to write about in February than love? Before I started writing this column, I asked what topics people ... Read more -
Cannabinol Identified as Active Component of Potential EB Treatment INM-755, InMed Announces
Cannabinol (CBN), a rare type of cannabinoid, is the active component of InMed Pharmaceuticals‘ INM-755 treatment candidate for epidermolysis bullosa (EB). CBN is also the ... Read more -
FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar
The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. ... Read more
Subscribe to our mailing list
Get regular updates delivered to your email box.
Thanks! Please check your email for a confirmation email.
-
Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
An abundance of events are afoot around the world to mark Rare Disease Day ... -
With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan ... -
Krystal Opens Second Facility to Support Production and Potential Commercialization of its Gene Therapy B-VEC
Krystal Biotech is opening its second gene therapy facility, called Astra, to support the ... -
Aegle Raises $4M to Fund First Phase 1/2 Trial of Experimental Therapy AGLE-102 for DEB
Aegle Therapeutics has raised $4 million to fund the first clinical trial evaluating AGLE-102, ... -
Abeona Launches Phase 3 Trial Testing EB-101 Cell Therapy for RDEB
Abeona Therapeutics has begun its Phase 3 VIITAL trial evaluating EB-101, a gene-corrected cell ... -
It’s My Body: Why Unsolicited Comments Can Be Hurtful
It’s annoying — yes, freaking annoying — when strangers and even acquaintances feel compelled ...
Factors Beyond COL7A1 Mutations May Contribute to Dystrophic EB Forms, Study Suggests
News
A study from Spain has raised the possibility that the development of dystrophic epidermolysis bullosa (DEB) could be attributable to additional factors besides mutations in the COL7A1 gene. Researchers reached this conclusion after studying three unrelated patients with different forms of ... Read more